HARMONY JPM 2025FINAL - Website.pdf

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1、Copyright 2025 Harmony Biosciences.All rights reserved.43rd AnnualJP Morgan Healthcare ConferenceJ E F F R E Y M.D AY N O,M DJ a n u a r y 1 5,2 0 2 5|S a n F r a n c i s c oForward-Looking StatementsThis presentation includes forwardlooking statements within the meaning of the Private Securities Li

2、tigation Reform Act of 1995.All statements other than statements of historical facts contained in these materials or elsewhere,including statements regarding Harmony Biosciences Holdings,Inc.s(the“Company”)future financial position,preliminary financial results,business strategy and plans and object

3、ives of management for future operations,should be considered forward-looking statements.Forward-looking statements use words like“believes,”“plans,”“expects,”“intends,”“will,”“would,”“anticipates,”“estimates,”“may,”“could,”“might,”“continue,”“potential,”and similar words or expressions in discussio

4、ns of the Companys future operations,financial performance or the Companys strategies,but the absence of these words does not mean that a statement is not forward-looking.These statements are based on current expectations or objectives that are inherently uncertain.These forward-looking statements i

5、nvolve significant risks and uncertainties that could cause the actual results to differ materially from the expressed or implied forwarding-looking statements,including,but not limited to the risk factors discussed under the caption“Risk Factors”in the Companys Annual Report on Form 10-K filed with

6、 the U.S.Securities and Exchange Commission(the“SEC”)on February 22,2024 and its other filings with the SEC.While the Company may elect to update such forward-looking statements at some point in the future,it disclaims any obligation to do so,even if subsequent events cause its views to change.This

7、presentation includes information related to market opportunity as well as cost and other estimates obtained from internal analyses and external sources.The internal analyses are based upon managements understanding of market and industry conditions and have not been verified by independent sources.

8、Similarly,the externally sourced information has been obtained from sources the Company believes to be reliable,but the accuracy and completeness of such information cannot be assured.Neither the Company,nor any of its respective officers,directors,managers,employees,agents,or representatives,(i)mak

9、e any representations or warranties,express or implied,with respect to any of the information contained herein,including the accuracy or completeness of this presentation or any other written or oral information made available to any interested party or its advisor(and any liability therefore is exp

10、ressly disclaimed),(ii)have any liability from the use of the information,including with respect to any forward-looking statements,or(iii)undertake to update any of the information contained herein or provide additional information as a result of new information or future events or developments.This

11、 presentation concerns product candidates that are or have been under clinical investigation and which have not yet been approved for marketing by the U.S.Food and Drug Administration,European Medicines Agency or other foreign regulatory authorities.These product candidates are currently limited by

12、U.S.Federal law to investigational use,and no representations are made as to their safety or effectiveness for the purposes for which they are being investigated.Preliminary Financial ResultsThe preliminary financial results of the Company for the fiscal year ended December 31,2024 included in this

13、presentation are estimates and represent the most current information available to the Companys management,as financial closing procedures for the fourth quarter and fiscal year ended December 31,2024 are not yet complete.The Company expects that its actual results to be reported in its Annual Repor

14、t on Form 10-K for the year ended December 31,2024 will not differ materially from the preliminary results,however,these results are subject to change following the completion of year-end accounting procedures and adjustments,including the execution of the Companys internal control over financial re

15、porting,the completion of the preparation and audit of the Companys financial statements and the subsequent occurrence or identification of events prior to the formal issuance of the audited financial statements for fiscal year 2024.Harmony Biosciences|J.P.Morgan Healthcare Conference2January 15,202

16、5INNOVATIVEPATIENT-FOCUSEDPROFITABLE BIOTECHCATALYST-RICH PIPELINEHarmony Biosciences|J.P.Morgan Healthcare Conference4January 15,20254 Key Catalysts One every Q in 2025$1B+Potential opportunity for WAKIX in narcolepsy$3B+Establishing leadership position in CNS6 Phase 3Development programs by year e

17、nd 2025 MULTIPLE MILESTONES IN2025DURABLE LONG-TERM VALUE CREATIONSLEEP/WAKENEUROBEHAVIORALEPILEPSYEACH FRANCHISE WITH POTENTIAL Peak Sales of$1-2BHarmony Biosciences|J.P.Morgan Healthcare Conference5January 15,2025Innovative Late-Stage PipelineHarmony Biosciences|J.P.Morgan Healthcare Conference6Ja

18、nuary 15,2025PRODUCT/INDICATIONPRE-INDPHASE 1PHASE 2PHASE 3REGULATORY FILINGMARKETED PRODUCTSleep/WakeWAKIXEDS in Narcolepsy(Adults)Cataplexy in Narcolepsy(Adults)EDS in Narcolepsy(Pediatric)PitolisantIdiopathic Hypersomnia(IH)Prader-Willi Syndrome(PWS)Myotonic Dystrophy(DM1)Pitolisant Gastro-Resist

19、ant(GR)Pitolisant High-Dose(HD)BP1.15205(Orexin-2 Receptor Agonist)Sleep/Wake DisordersHBS-102PWSNeurobehavioralZYN-002(Cannabidiol Gel)Fragile X Syndrome(FXS)22q11.2 Deletion Syndrome(22q)Rare EpilepsyEPX-100(Clemizole Hydrochloride)Dravet Syndrome(DS)Lenox-Gastaut Syndrome(LGS)EPX-200(Lorcaserin)D

20、evelopmental and Epileptic Encephalopathies(DEE)3CNSFRANCHISES13DEVELOPMENT PROGRAMS8ASSETS6PHASE 3 PROGRAMS BY YEAR ENDWAKIX Is One of the Most Successful Orphan/Rare Launches With Demonstrated Durable Revenue Generation Harmony Biosciences|J.P.Morgan Healthcare Conference7January 15,2025WAKIX Net

21、Revenue Growth 20202024$160$305$438$582$714$0$200$400$600$80020202021202220232024Net Revenue($M)1KEY TAKEAWAY1.Net Revenue for fiscal year 2024 is preliminary,unaudited and subject to change 2.https:/narcolepsynetwork.org/accessed Feb 202480,00090,000 50%of Patients Undiagnosed170,000Diagnosed Narco

22、lepsy PatientsUndiagnosed Narcolepsy PopulationPeople Living With Narcolepsy in the U.SSustained,durable revenue for WAKIX over 5 years on the market2025 Net Revenue Guidance Harmony Biosciences|J.P.Morgan Healthcare Conference8January 15,2025ChrisLiving with NarcolepsyKEY TAKEAWAY$820M-$860MNET REV

23、ENUE GUIDANCEWAKIX represents a potential$1B+opportunity in narcolepsy alone2025 Anticipated CatalystsHarmony Biosciences|J.P.Morgan Healthcare Conference9January 15,20251Q25Pitolisant IH sNDAFDA decision on file acceptance(potential approval in 2025)2Q25BP1.15205(OX2R agonist)Preclinical data prese

24、ntation at SLEEP 2025 IMPD submission(IND submission 2H)3Q25ZYN002FXS Phase 3 topline data readout Pitolisant-GR Pivotal BE study readout4Q25Pitolisant-HD initiation of pivotal Phase 3 trial in NarcolepsyLate-stage pipeline driving a catalyst-rich 2025KEY TAKEAWAYTOTALITY OF DATA FROM THE PHASE 3 IN

25、TUNE STUDYOpen-label,randomized withdrawal and long-term extension dataREAL WORLD AND CLINICAL EXPERIENCE DATAData from compassionate use program and top KOL clinical experience FAVORABLE BENEFIT/RISK PROFILEUnmet medical need/unique safety profileVOICE OF THE PATIENT REPORTOutput from Externally-Le

26、d Patient-focused Drug Development meeting with active FDA participationESTABLISHED SAFETYNon-scheduled and simple dosing regimenQ1 Catalyst:Pitolisant for Idiopathic Hypersomnia(IH)Harmony Biosciences|J.P.Morgan Healthcare Conference10January 15,2025FDA decision on IH sNDA file acceptance(potential

27、 approval in 2025)024681012141618Strong and Durable Improvement in EDS in Patients With IH(as Measured by ESS)Harmony Biosciences|J.P.Morgan Healthcare Conference11January 15,2025The mean ESS Score stayed within the normal level of wakefulness throughout the long-term extension periodKEY TAKEAWAYNOR

28、MAL LEVEL OF WAKEFULNESS Start of StudyEnd of 8 WeeksPitolisant RWDPlaceboMean ESS ScoreDAY 22MONTH 7MONTH 13Open Label Extension(52 weeks)Randomized Withdrawal Phase(4 Weeks)Open Label Phase(8 Weeks)9.4 POINTIMPROVEMENT SUSTAINED RESPONSE WITHIN NORMAL RANGE UP TO 13 MONTHSITT population:Data on fi

29、le0%10%20%30%40%50%60%70%80%90%100%Percentage of Pitolisant Patients within the Normal Level of WakefulnessHarmony Biosciences|J.P.Morgan Healthcare Conference12January 15,2025Pitolisant demonstrates high patient response rates in maintaining normal level of wakefulness observed through 13 months of

30、 treatmentKEY TAKEAWAYStart of StudyEnd of 8 WeeksPitolisant RWDPlaceboDAY 22MONTH 7MONTH 13Open Label Extension(52 weeks)Randomized Withdrawal Phase(4 Weeks)Open Label Phase(8 Weeks)78.42%73.9%70.3%75.6%of Patients on Pitolisant with ESS 600 x9800 x 2000 x3000 x 5000 xNot reportedNot reportedNot re

31、portedDosing regimenPotential for once-daily oral dosingOnce a day dosingNot reported Twice a day dosingIV dosingTwice a day dosingOnce a day dosing Not reportedBP1.15205:Most potent OX2R Agonist(In Vitro Pharmacology Data)Harmony Biosciences|J.P.Morgan Healthcare Conference14January 15,2025The most

32、 potent orexin-2 receptor agonist(based on publicly available data)KEY TAKEAWAY1.Bioprojet/Harmony data on file;2.Lack et al.,World Sleep 2023,abstract;3.Hatanaka et al.,ACNP 2022,poster;4.Kimura et al.,World Sleep 2023,abstract;5.Yukitake et al.,Pharmacol Biochem Behav.2019,publication;6.Clinicaltr

33、ials.gov.Orexin-2 Receptor Agonist:Distinct Chemical Structure with Unique Properties and Potential Clinical BenefitsHarmony Biosciences|J.P.Morgan Healthcare Conference15January 15,2025Potential best-in-class OX2R agonist with possibility for broad clinical utilityKEY TAKEAWAYCombination of high po

34、tency and excellent selectivity translates to potentially low dose(s)for efficacy which provides dosing flexibility across all central disorders of hypersomnolence Selectivity Excellent selectivity in the context of potency that is several orders of magnitude higher compared to other OX2R agonists O

35、ver 140-fold selectivity over OX1R based on highest anticipated human dose from pre-clinical efficacy data Over 1000-fold selectivity against 150 other targets of interestHighest potency compared to other OX2R agonists to-dateExcellent PotencyandQ3 Catalyst:ZYN002 for the Treatment of Fragile X Synd

36、romeHarmony Biosciences|J.P.Morgan Healthcare Conference16January 15,2025Topline Data Readout from Pivotal Phase 3 RECONNECT TrialZYN002:INNOVATIVE PRODUCT PROFILEPurely synthetic cannabidiol(CBD);devoid of THCLEAD PROGRAM IN FRAGILE X SYNDROME(FXS)Additional opportunity in related disorder,22q dele

37、tion syndrome(22q)MARKET OPPORTUNITY80,000 patients in the US with FXS;similar for 22q,Worldwide rightsVERY HIGH UNMET NEEDNo approved products for FXS or 22qWould be first approved treatment for patients with FXSPhase 2 CONNECT Trial1:Completely Methylated Subgroup Harmony Biosciences|J.P.Morgan He

38、althcare Conference17January 15,2025Key secondary outcomesSOCIAL INTERACTION63%vs 37%(p=0.005*)IRRITABLE/DISRUPTIVE BEHAVIORS54%vs 33%(p=0.027*)SOCIAL AVOIDANCE(3 POINTS)56%vs 37%(p=0.030*)Clinically meaningful improvement in patients with complete methylationKEY TAKEAWAY40%MEDIAN IMPROVEMENT in soc

39、ially avoidant behaviors(p=0.027*)1.Berry-Kravis et al 2022*nominal statistical significanceOpen-Label Extension Patients on ZYN002Sustained Improvement in Patients With Complete Methylation of FMR1aHarmony Biosciences|J.P.Morgan Healthcare Conference18January 15,2025Change in ABC-CFXS Social Avoida

40、nce CONNECT-FXDouble-BlindbRECONNECTPopulationContinued improvement through 4 months used to support increasing treatment period in RECONNECT to 4 months-6-5-4-3-2-10012345678910111213141516171819202122232425262728Change from baselinecPlacebo(n)ZYN002(n)4847474549464543434436403133313310141519MonthZ

41、YN002PlaceboSwitch from Placebo to ZYN002a.Patients matching primary efficacy population in RECONNECT.b.ZYN2-CL-016(CONNECT-FX).c.Least square mean SE;reduction equals improvement.Pivotal Phase 3 RECONNECT Trial:Design Optimized from Phase 2 CONNECT-FX TrialHarmony Biosciences|J.P.Morgan Healthcare

42、Conference19January 15,2025Successful completion of Phase 3 pivotal trial expected to satisfy requirements for an NDA submission in the U.S.and a marketing authorization application in the EU.Primary endpoint:Patients with complete methylationExtended treatment period to 16 weeksIncreased dose for i

43、ndividuals 50 kgMore patient and family friendlyLearnings from Connect-FX trial informed RECONNECT study designKEY TAKEAWAYQ4 Catalyst:Pitolisant-HD in NarcolepsyHarmony Biosciences|J.P.Morgan Healthcare Conference20January 15,2025Initiation of pivotal Phase 3 trial of Pitolisant-HD in narcolepsy DE

44、SIGNED TO ADDRESS THE LARGEST UNMET MEDICAL NEED IN NARCOLEPSY Providing greater efficacy for both excessive daytime sleepiness and cataplexyPRELIMINARY IP FILED TO EXTEND PITOLISANT FRANCHISE INTO 2040S PROGRAM TO INCLUDE NOVEL ENDPOINT TO ASSESS NARCOLEPSY-RELATED FATIGUEPitolisant Franchise Poise

45、d to Drive Durable Patient and Revenue Growth to the Mid-2040s Two meaningfully differentiated product profiles building off WAKIX with PDUFAs prior to LOE Provisional patents filed out to 2044 to extend durable patient and net revenue growth Pursuing other indications(IH,DM1)to drive incremental pa

46、tient,net revenue growthHarmony Biosciences|J.P.Morgan Healthcare Conference21January 15,2025*With pediatric exclusivity;on-track to meet the requirements for pediatric exclusivity WAKIX(LOE Q3 2030*)$1B+foundation in narcolepsy alonePITOLISANT-GR(PDUFA DATE 2026)Expands pitolisant patient base$300$

47、500M Opportunity PITOLISANT-HD(PDUFA DATE 2028)Further expands pitolisant patient base and extends pitolisant franchise$1B+opportunity in narcolepsy alone 20192026202820302040Extension of Pitolisant franchise beyond 2030 with enhanced product profiles Poised to deliver durable patient growth and sig

48、nificant revenue to the mid 2040sKEY TAKEAWAYEPX-100:Most Advanced and Differentiated 5HT2(serotonin)Agonist Development Program Harmony Biosciences|J.P.Morgan Healthcare Conference22January 15,2025EPX-100https:/pubchem.ncbi.nlm.nih.gov/compound/ClemizolePROVEN MoA Established serotonergic mechanism

49、 of action confirmed via highly predictive zebra-fish modelPROVEN SAFETYClemizole was marketed for 20 yrs with no significant safety and/or tolerability signals from post marketing exposureEmerging safety and tolerability profile from the Phase 3 study in DS is promising;no need for special laborato

50、ry or cardiac monitoring requiredDOSING REGIMENBID dosing;very important clinical consideration for patients with DEEsADVANCED CLINICAL DEVELOPMENTActively enrolling patients in US and EU in a phase 3 registrational study in Dravet syndrome;Topline data in 2026Initiated Phase 3 registrational study

51、in LGS in Q4 2024;Topline Data in 2026Catalyst-Rich Pipeline Driving Value Beyond 2025Harmony Biosciences|J.P.Morgan Healthcare Conference23January 15,20252026ZYN002 FXS PDUFAPitolisant-GR PDUFAEPX100 DS/LGS Phase 3 topline data(TLD)Pitolisant PWS Phase 3 TLDOX2R SAD/MAD PK readouts2027 2028Pitolisa

52、nt-HD Phase 3 TLD in narcolepsy(2027)Pitolisant-HD PDUFA for narcolepsy(2028)ZYN002 22q deletion syndrome Phase 3 TLDEPX100 DS/LGS PDUFAPitolisant PWS PDUFAPipeline poised to deliver one or more new product or indication launches each year over the next 4 years KEY TAKEAWAYHarmony Biosciences|J.P.Mo

53、rgan Healthcare Conference24January 15,20254 Key Catalysts One every Q in 2025$1B+Potential opportunity for WAKIX in narcolepsy$3B+Establishing leadership position in CNS6 Phase 3Development programs by year end 2025 MULTIPLE MILESTONES IN2025DURABLE LONG-TERM VALUE CREATIONcompany/harmonybiosciences/harmony_biosciencesharmonybiow w w.h a r m o n y b i o s c i e n c e s.c o m